NCT02725567

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation

A Phase 3, 2 Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 57 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 1 Month – 24 Months
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in participants with cystic fibrosis (CF) who are \<24 months of age at treatment initiation and have an ivacaftor-responsive CF transmembrane conductance regulator (CFTR) gene mutation.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	IVA	Granules in sachet for oral administration.

Timeline

• Start date: 2016-06-02
• Primary completion: 2022-06-28
• Completion: 2022-06-28
• First posted: 2016-04-01
• Last updated: 2023-09-07
• Results posted: 2023-09-07

Locations

23 sites across 5 countries: United States, Australia, Canada, Ireland, United Kingdom